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About the Regulatory Profession

The regulatory function is vital in making safe and effective healthcare products available worldwide. Individuals who ensure regulatory compliance and prepare submissions, as well as those whose main job function is clinical affairs or quality assurance are all considered regulatory professionals.

Regulatory Code of Ethics

One of our most valuable contributions to the profession is the Regulatory Code of Ethics. The Code of Ethics provides regulatory professionals with core values that hold them to the highest standards of professional conduct.

Regulatory Competency Framework

Like all professions, regulatory is based on a shared set of competencies. The Regulatory Competency Framework describes the essential elements of what is required of regulatory professionals at four major career and professional levels.

Regulatory Convergence

Join the brightest minds in regulatory at the annual Regulatory Convergence. See the global regulatory community in action. Intensive workshops. Topical sessions. Meet ups with regulators. This is where it all comes together.

California Gov. Jerry Brown (D) on Sunday vetoed a bill that would've allowed a pharmaceutical company to make an investigational drug available to a patient with a terminal illness on the recommendation of two physicians.

Brown explained in a message with his decision that the so-called "Right to Try" (RTT) legislation is intended to help patients with life-threatening conditions, and in some cases these patients can already gain access to experimental treatments under the US Food and Drug Administration's (FDA) compassionate use program.

"Before authorizing an alternative state pathway, we should give this federal expedited process a chance to work," Brown said.

Do the Laws Work?

Since 2014, RTT laws have been adopted in 24 states, in addition to federal RTT legislation that was introduced in July, in the hopes of allowing access to potentially life-saving treatments more easily. The laws are modeled off a federal policy known as "Compassionate Use," but contain several key changes meant to make it faster and easier for patients to obtain experimental therapies.

However, more recently, a number of questions have been raised as to whether these bills have actually brought experimental treatments to the patients in need, and whether they're even necessary because of a more simplified FDA compassionate use program.

Dr. Arthur Caplan, director of the Division of Medical Ethics at New York University's Langone Medical Center, who's also part of the NYU Working Group on Compassionate Use and Pre-Approval Access, told reporters in late August: "It's fair to say we haven't yet found a case where right to try laws have facilitated access to anything."

Caplan and several others also previously wrote in Health Affairs that although the "laws have created an expectation that terminally ill patients will be able to quickly access potentially life-saving treatments by being exempted" from FDA rules, "this expectation is, quite simply, false.

"Putting aside the invalidity of RTT laws [because of federal law preempting state law] and the false hope they create, these laws also fail to address more fundamental health and safety issues. RTT laws seek to grant vulnerable patients access to unproven and potentially harmful drugs or other medical products without any expert safeguards, such as scientific or ethical review by an Institutional Review Board or similar body," Caplan and others wrote.

FDA Action

Brown and Caplan's comments come as in February, FDA proposed draft guidance that would streamline the procedures for its compassionate use program and make it easier for patients in need to obtain the drugs they're seeking more quickly via their physicians.

And if the application process proves to still be too slow for a particular patient, FDA notes that in an "emergency situation that requires the patient to be treated before a written submission can be made, the request to use the investigational drug for individual patient expanded access may be made by telephone (or other rapid means of communication) to the appropriate FDA review division, and authorization of the emergency use may be given by the FDA official over the telephone," provided the physician explains how the use of the experimental drug meets the statutory requirements and a written submission is sent to FDA within 15 working days of the authorization to use the drug.

And unlike the RTT legislation, the FDA compassionate use program has brought experimental drugs to patients in need. One recent example from earlier this month involved a 10-year-old Michigan boy with a brain tumor who sought "compassionate use" of an AbbVie drug and received clearance to use the drug from FDA and an institutional review board within 10 days.